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A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)

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ClinicalTrials.gov Identifier: NCT02628743
Recruitment Status : Completed
First Posted : December 11, 2015
Last Update Posted : March 8, 2019
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Tracking Information
First Submitted Date  ICMJE December 1, 2015
First Posted Date  ICMJE December 11, 2015
Last Update Posted Date March 8, 2019
Actual Study Start Date  ICMJE January 20, 2016
Actual Primary Completion Date February 19, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 9, 2017)
Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs) [ Time Frame: Baseline up to approximately 5 years ]
Original Primary Outcome Measures  ICMJE
 (submitted: December 9, 2015)
  • Incidence of adverse events [ Time Frame: Up to 4 years ]
  • Safety laboratory tests, Vital signs and electrocardiogram parameters [ Time Frame: Up to 4 years ]
Change History Complete list of historical versions of study NCT02628743 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 9, 2017)
  • Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Apparent Clearance (CL/f) of Olesoxime [ Time Frame: Pre-dose (Hour 0) at Weeks 1, 13, 26, 39, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Apparent Volume of Distribution at Steady State (Vss/F) of Olesoxime [ Time Frame: Pre-dose (Hour 0) at Weeks 1, 13, 26, 39, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Pediatric Quality of Life Questionnaire (PedsQL) Generic Core Scale Version 4.0 Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • PedsQL Neuromuscular Module Version 3.0 Scale Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver PedsQL Generic Core Scales Version 4.0 Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver PedsQL Neuromuscular Module Version 3.0 Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • EQ-5D-5L Visual Analogue Scale (EQ-5D-5L VAS) Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver Proxy EQ-5D-5L Questionnaire Index Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver Proxy EQ-5D-5L VAS Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Work Productivity and Activity Impairment: Caregiver (WPAI:CG) Questionnaire Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
  • Caregiver Generic Health Related Quality of Life: Short Form-36 Questionnaire Score [ Time Frame: Baseline (Week 1), Weeks 26, 52, 78, 104, 130; thereafter every 26 weeks up to 5 years ]
Original Secondary Outcome Measures  ICMJE
 (submitted: December 9, 2015)
  • Change from baseline in Motor Function Measure (MFM) D1+D2 +D3 score and total score [ Time Frame: Baseline (Week 1), Week 26, Week 52, Week 104, thereafter every 26 weeks up to 4 years ]
  • Change from baseline in Hammersmith Functional Motor Scale (HFMS) score [ Time Frame: Screening (Day -42 to Day -1), Week 13, Week 39, Week 78, Week 130, thereafter every 26 weeks up to 4 years ]
  • Predose (trough) plasma olesoxime concentration at each visit [ Time Frame: Week 1, Week 13, Week 26, Week 52, Week 104, thereafter every 12 months up to 4 years ]
  • The Pediatric Quality of Life Inventory (PedsQL) core scales and neuromuscular sub-scales [ Time Frame: Week 1, Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 26 weeks up to 4 years ]
  • The EuroQol 5-Dimension Questionnaire, 5-level version (EQ-5D-5L) score [ Time Frame: Week 1, Week 26, Week 52, Week 78, Week 104, Week 130, thereafter every 26 weeks up to 4 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
Official Title  ICMJE Multicenter, Open-Label, Single-Arm Study to Evaluate Long-Term Safety, Tolerability, and Effectiveness of 10 mg/kg BID Olesoxime in Patients With Spinal Muscular Atrophy
Brief Summary The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of the following two clinical studies: TRO19622 CL E Q 1115-1 (NCT ID XXX, open-label Phase Ib, multicenter, single- and multiple- dose study) or TRO19622 CL E Q 1275-1 (NCT01302600, Phase II/III, adaptive, parallel-group, double blind, randomized, placebo-controlled, multicenter, multinational study).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Muscular Atrophy, Spinal
Intervention  ICMJE Drug: Olesoxime
Participants will receive homogeneous suspension of olesoxime.
Study Arms  ICMJE Experimental: Olesoxime

Participants who have consented to the dose increase will receive 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube with breakfast and dinner, preferably at the same time of the day throughout the study. If the drug administration does not coincide with one of the scheduled meals, a snack should be taken prior to drug administration. Preferably there should be at least 10 hours between the morning and evening dose. The total dose in this study will not exceed 2000 mg.

Participants who do not consent to the dose increase will continue with the previous dosage and receive a dose of 10 mg/kg suspension once a day orally or via a naso-gastric or gastronomy tube with the main meal, preferably at the same time of the day.

Intervention: Drug: Olesoxime
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 7, 2017)
131
Original Estimated Enrollment  ICMJE
 (submitted: December 9, 2015)
171
Actual Study Completion Date  ICMJE February 19, 2019
Actual Primary Completion Date February 19, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Participation in the previous studies (TRO19622 CL E Q 1115-1 or TRO19622 CL E Q 1275-1)
  • For women of childbearing potential: agreement to use an acceptable birth control method during the treatment period and for at least 28 days after the last dose of olesoxime

Exclusion Criteria:

  • Female participants who are pregnant or lactating, or intending to become pregnant during the study
  • Participants who, in the opinion of the investigator, are not suitable to participate in this open-label study
  • Participants who have developed study drug hypersensitivity to olesoxime or one of the formulation excipients, including sesame oil
  • Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening
  • Concomitant or previous participation in a survival motor neuron 2 (SMN2) targeting antisense oligonucleotide study within 6 months prior to screening
  • History of human immunodeficiency virus infection, history of Hepatitis B infection within the past year, history of Hepatitis C infection which has not been adequately treated
  • History of illicit drug or alcohol abuse within 12 months prior to screening, in the investigator's judgment
  • Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the participant's safe participation in and completion of the study
  • History or presence of an abnormal electrocardiogram (ECG) that is clinically significant in the investigator's opinion
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   France,   Germany,   Italy,   Netherlands,   Poland,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02628743
Other Study ID Numbers  ICMJE BN29854
2015-001589-25 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Hoffmann-La Roche
Study Sponsor  ICMJE Hoffmann-La Roche
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Trials Hoffmann-La Roche
PRS Account Hoffmann-La Roche
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP